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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02384460




Registration number
NCT02384460
Ethics application status
Date submitted
13/02/2015
Date registered
10/03/2015
Date last updated
9/04/2020

Titles & IDs
Public title
ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
Scientific title
A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa
Secondary ID [1] 0 0
2014-002288-14
Secondary ID [2] 0 0
SD-005
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Epidermolysis Bullosa 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Skin 0 0 0 0
Dermatological conditions
Skin 0 0 0 0
Other skin conditions

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SD-101-6.0 cream
Treatment: Drugs - Placebo (SD-101-0.0) cream

Experimental: SD-101-6.0 cream - SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days

Placebo Comparator: Placebo (SD-101-0.0) cream - SD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days


Treatment: Drugs: SD-101-6.0 cream
applied topically once a day for 90 days

Treatment: Drugs: Placebo (SD-101-0.0) cream
applied topically once a day for 90 days

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time To Complete Target Wound Closure Within 3 Months - Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. Time to target wound closure was measured from the date of the first administration of the study drug to the date of target wound closure. Participants were censored if they did not have a response within 3 months, or withdrew earlier before the confirmation of their target wound closing. This primary end point displays the mean time to complete target wound closure, analyzed using a Kaplan-Meier approach.
Timepoint [1] 0 0
From baseline to Month 3 visit
Primary outcome [2] 0 0
The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months - Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. Participants were considered responders if they experienced complete wound closure at the Week 2 or Months 1, 2, or 3 visits. If a target wound was documented to have closed at a given visit, it was considered closed at all subsequent visits. This primary end point displays the percentage of participants from the ITT population who had complete target wound closure by the end of the study period (that is, 3 months). Analysis was performed on participants with post-baseline wound closure data.
Timepoint [2] 0 0
From baseline to Month 3 visit
Secondary outcome [1] 0 0
Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits - Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. The percentage of participants who completed target wound closure at the Month 1 and Month 2 study visits is displayed. If a target wound was documented to have closed at a given visit, it was considered closed at all subsequent visits.
Timepoint [1] 0 0
From baseline to Month 1 and Month 2 visits
Secondary outcome [2] 0 0
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit - Lesional skin was defined as areas that contained any of the following: blisters, erosions, ulcerations, scabbing, bullae, or eschars, as well as areas that were weeping, sloughing, oozing, crusted, or denuded. BSAI was calculated as a percentage, ranging from 0% to 100%, of affected body surface area, recorded for each defined body region (that is, head/neck, upper limbs, trunk [includes groin], and lower limbs), multiplied by the weighting factor, then summed for all body regions.
Timepoint [2] 0 0
Baseline, Month 3 visit
Secondary outcome [3] 0 0
Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit - Total body wound burden was calculated using BSAI. A wound defined as an open area on the skin (that is, epidermal covering disrupted). BSAI was calculated as a percentage, ranging from 0% to 100%, of affected body surface area, recorded for each defined body region (that is, head/neck, upper limbs, lower limbs, trunk [includes groin]), and multiplied by the weighting factor, then summed for all body regions.
Timepoint [3] 0 0
Baseline, Month 3 visit
Secondary outcome [4] 0 0
Change From Baseline In Itching Score At Day 7 - Itching was assessed using the 5-point Itch Man Pruritus Assessment Tool. For participants up to 5 years of age, itching was assessed using caretaker's response and participants 6 years of age and older self-reported their itching assessments based on the following scores: 0=Comfortable, no itch; 1=itches a little, does not interfere with activity; 2=itches more, sometimes interferes with activity; 3=itches a lot, difficult to be still, concentrate; 4=itches most terribly, impossible to sit still or concentrate. Itching scores were categorized into 3 groups based on improvement; Improved or No Itching, Not Improved, and Missing. An itching score reduction from baseline greater than or equal to 1 point on the scale was classed as improved.
Timepoint [4] 0 0
Baseline, Day 7
Secondary outcome [5] 0 0
Change From Baseline In Pain Score At Day 7 - Change in pain assessed at Day 7 compared to baseline was measured using the Face, Legs, Activity, Cry, and Consolability (FLACC) behavioral scale for participants 1 month to 3 years of age. Each of the 5 FLACC categories was scored from 0 to 2, which resulted in a total score between 0 and 10 with 0=Relaxed and comfortable, 1 to 3=Mild discomfort, 4 to 6=Moderate pain, and 7 to 10=Severe discomfort/pain. For participants 4 years of age and older, the "Wong Faces Pain Scale" was used. This scale shows a series of faces ranging from a happy face at 0, which represents "no hurt," to a crying face at 10, which represents "hurts worst". Pain scores were categorized into 3 groups based on improvement: Improved or No Pain, Not Improved, and Missing. A pain score reduction from baseline greater than or equal to 2 points on the scale was classed as improved.
Timepoint [5] 0 0
Baseline, Day 7

Eligibility
Key inclusion criteria
- Informed Consent form signed by the participant or participant's legal representative;
if the participant was under the age of 18 but capable of providing assent, signed
assent from the participant.

- Participant (or caretaker) must have been willing to comply with all protocol
requirements.

- Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.

- Participant must have had 1 target wound (size 10 to 50 cm^2) at study entry.

- Participants 1 month and older.

- Target wound must have been present for at least 21 days.
Minimum age
1 Month
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Participants who did not meet the entry criteria outlined above.

- Selected target wound did not have clinical evidence of local infection.

- Use of any investigational drug within the 30 days before enrollment.

- Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.

- Use of systemic or topical steroidal therapy within the 30 days before enrollment.
(Inhaled steroids and ophthalmic drops containing steroids were allowed).

- Use of systemic antibiotics within the 7 days before enrollment.

- Current or former malignancy.

- Arterial or venous disorder resulting in ulcerated lesions.

- Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at
screening and every 30 days until the final visit for female participants of
childbearing potential).

- Females of childbearing potential who were not abstinent and not practicing a
medically acceptable method of contraception.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
- Kogarah
Recruitment hospital [2] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment postcode(s) [2] 0 0
3050 - Parkville
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New Hampshire
Country [10] 0 0
United States of America
State/province [10] 0 0
New Jersey
Country [11] 0 0
United States of America
State/province [11] 0 0
New York
Country [12] 0 0
United States of America
State/province [12] 0 0
North Carolina
Country [13] 0 0
United States of America
State/province [13] 0 0
Ohio
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
South Carolina
Country [16] 0 0
United States of America
State/province [16] 0 0
Texas
Country [17] 0 0
United States of America
State/province [17] 0 0
Washington
Country [18] 0 0
Austria
State/province [18] 0 0
Salzburg
Country [19] 0 0
France
State/province [19] 0 0
Nice
Country [20] 0 0
France
State/province [20] 0 0
Paris
Country [21] 0 0
France
State/province [21] 0 0
Toulouse
Country [22] 0 0
Germany
State/province [22] 0 0
Freiburg
Country [23] 0 0
Germany
State/province [23] 0 0
Hannover
Country [24] 0 0
Israel
State/province [24] 0 0
Tel Aviv
Country [25] 0 0
Italy
State/province [25] 0 0
Milano
Country [26] 0 0
Lithuania
State/province [26] 0 0
Kaunas
Country [27] 0 0
Netherlands
State/province [27] 0 0
Groningen
Country [28] 0 0
Poland
State/province [28] 0 0
Koszykowa
Country [29] 0 0
Serbia
State/province [29] 0 0
Belgrade
Country [30] 0 0
Spain
State/province [30] 0 0
Madrid
Country [31] 0 0
United Kingdom
State/province [31] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Scioderm, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Amicus Therapeutics
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0)
cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding
Source - United States Food and Drug Administration (FDA) Office of Orphan Products
Development (OOPD).
Trial website
https://clinicaltrials.gov/show/NCT02384460
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor
Address 0 0
Amicus Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications